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Early trial data supports neurodegenerative disease-modifying drug

Drug Discovery World

All doses were well-tolerated and there were no serious adverse events. We believe that the results from the Phase Ib MAD study directly support larger scale Phase II clinical development to more fully understand the potential for ALX-001 to become the first-ever disease-modifying small molecule for neurodegenerative diseases.”

Disease 130
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Molecular photoswitch improves visual acuity in retinitis pigmentosa

Drug Discovery World

Topline results from a Phase I/II clinical trial have shown that Kiora’s light-restoring small molecule KIO-301 has the potential to meaningfully improve vision in patients with retinitis pigmentosa (RP). This new technology offers hope to patients living with late stage inherited retinal diseases.

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Sosei Heptares and Biohaven Enter Global Collaboration and License Agreement to Advance Novel Small-Molecule CGRP Antagonist Portfolio

The Pharma Data

Biohaven acquires exclusive global rights to a portfolio of novel, small-molecule CGRP antagonists. The lead candidate, HTL0022562, has advanced through preclinical development demonstrating promising and differentiated properties for further investigation in human trials. Vlad Coric , M.D.,

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Sosei Heptares’ COVID-19 Program Identifies Potent Broad Spectrum Anti-Viral Small Molecules for Further Development

The Pharma Data

The Company is also aiming to leverage the highly conserved structure of the SARS-CoV-2 M pro protease as a basis for the design of novel oral small molecules against predicted future variants of SARS-CoV-2 and other related human viruses. View original content: [link]. SOURCE Sosei Heptares. Source link.

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Sentiment & Themes Emerging From JPM 2024

LifeSciVC

The market for each target class likely asymptotes with the number of Pharma or large biotech who can clinically develop and commercialize such assets; thus, there is intense focus on the first handful of assets to market. Small molecule GLP1s?

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Comment: LSX World Congress 2023 

Drug Discovery World

DDW’s Megan Thomas comments on the event. New approaches to partnering In this event in the Biotech track, Clive Cookson, Science Editor at the Financial Times, interviewed John McDonald, Corporate Vice President, Global R&D Business Development, Novo Nordisk.

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Positive Phase I results for first-in-kind small molecule complement inhibitor

Drug Discovery World

ANX1502 is a first-in-kind oral, selective small molecule inhibitor that targets the active form of C1s responsible for propagating classical pathway activation in association with C1q. Across all doses evaluated, ANX1502 was generally well tolerated with mild to moderate treatment-emergent adverse events (TEAEs).